A Dose-Optimization Study of Aldurazyme┬« (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Information source: Genzyme
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome
Intervention: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Drug)
Phase: Phase 4
Sponsored by: Genzyme
The main purpose of this study is to evaluate differences in the pharmacodynamic response of
4 Aldurazyme« (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I)
disease through measuring urinary GAG levels, liver volume, and functional tests (6-minute
Official title: A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme« (Laronidase) in Patients With Mucopolysaccharidosis I
Study design: Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study
Primary outcome: To evaluate differences in the pharmacodynamic response and safety of Aldurazyme« (laronidase) does regimens
Minimum age: N/A.
Maximum age: N/A.
- Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and
symptoms of MPS I, and a documented fibroblast or leukocyte ╬▒-L-iduronidase enzyme
activity level of less than 10% of the lower limit of the normal range of the
- Weigh at least 12. 5 kg.
- Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate
- Provide signed, written informed consent prior to any protocol-related procedures
being performed. Consent of a legally authorized guardian(s) is (are) required for
patients under 18 years. If the patient is under 18 years old and can understand the
consent, written informed consent will be required from both the patient and the
- Have previously received Aldurazyme┬« (laronidase).
- Have a suspected hypersensitivity to Aldurazyme┬« (laronidase) or known
hypersensitivity to components of the infusion solution.
- Have previously undergone hematopoietic stem cell transplantation (HSCT; i. e., from
bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ
- Have a medical condition, serious inter-current illness, or other extenuating
circumstance that may interfere with study compliance including all prescribed
evaluations and follow-up activities, except the 6MWT.
Note: All patients may not be capable of performing the 6MWT due to age and/or maturity
level. Exemption from performing the 6MWT must be obtained in writing by the investigator
from the sponsor's medical monitor prior to enrollment.
- Have an acute illness that requires surgical intervention, and/or anticipates surgery
during study participation, and/or has had surgery within 30 days prior to study
- Have received an investigational drug within 30 days prior to study enrollment.
- Is pregnant or lactating. Female patients of childbearing potential must have a
negative pregnancy test [urine ╬▓-human chronic gonadotropin (hCG)] at entry (prior to
the first infusion). Note: All female patients of childbearing potential and sexually
mature males must be advised to use a medically accepted method of contraception
throughout the study.
Locations and Contacts
Hospital de Clinical de Porto Alegre, Porto Alegre CEP 90035-003, Brazil
Universidade Federal de Sao Paulo, San Paulo CEP 04023-062, Brazil
Hospital das Clinicas de Faculdade de Medicina da Universidade Federal de Minas Gerais, Belo Horizonte CEP 30130-100, Brazil
Division of Clinical and Metabolic Genetics, Toronto, Ontario M5G 1X8, Canada
Hospital Infantil Joana de Gusmao, Florianopolis, Santa Catarina CEP 88025-601, Brazil
US FDA Approved Full Prescribing Information for Aldurazyme┬«
Starting date: April 2004
Ending date: January 2006
Last updated: December 14, 2007